Intrathecal Baclofen Infusion for Reflex sympathetic Dystrophy related dystonia
| ISRCTN | ISRCTN43633981 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN43633981 |
| Secondary identifying numbers | P01.098; NTR403 |
- Submission date
- 26/02/2007
- Registration date
- 26/02/2007
- Last edited
- 15/10/2008
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Signs and Symptoms
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Prof J J van Hilten
Scientific
Scientific
Leiden University Medical Centre (LUMC)
Department of Neurology
Postzone K-05Q
P.O. Box 9600
Albinusdreef 2
Leiden
2300 RC
Netherlands
| Phone | +31 (0)71 526 6065 |
|---|---|
| J.J.van_Hilten@lumc.nl |
Study information
| Study design | Part one: a single blinded placebo-run-in, dose-escalation design Part two: thereafter, in responders, an open trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Single-centre |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Scientific title | |
| Study acronym | The BIRD study |
| Study objectives | Dystonia associated with reflex sympathetic dystrophy responds markedly to intrathecal baclofen (ITB). |
| Ethics approval(s) | Approval received from the local ethics committee (Commissie Medische Ethiek) on the 2nd November 2001 (ref: P01.098). |
| Health condition(s) or problem(s) studied | Reflex sympathetic dystrophy (RSD) |
| Intervention | Screening phase: To evaluate the efficacy of ITB a percutaneous catheter is introduced into the lumbar subarachnoid space. All patients will start with a two-day placebo run-in, followed by a gradual titration of continuous intrathecal baclofen through an external pump. The daily baclofen dose will be increased according to a fixed schedule (200, 250, 300, 375, 450, 500, 600, 700 and 800 µg/24 hours). Depending on the response, the duration of the screening procedure may vary from one to two weeks. If a baclofen-related side-effect occurs at a particular dose, then depending on the severity of the side-effect the pump will be stopped or adjusted to a lower infusion rate. The outcome that is evaluated to determine if a patient will be implanted is the difference in change between Global Dystonia Severity (GDS - Visual Analogue Scale on which symptom severity is rated from zero [absent] to ten [most severe]) on ITB and placebo days. This difference is calculated through the following steps: 1. GDSbaclo: for each ITB day the sumscore of six one-hour intervals (11.00 a.m. - 4.00 p.m.) is determined. Likewise, for the two placebo days a mean placebo-sumscore is calculated (GDSplacebo) 2. GDShome: a similar mean sumscore of six one-hour intervals of the GDS at home is determined 3. For each day the GDS change score is calculated as follows: GDSbaclo - GDShome = GDSchangescore1, expressed in % (calculated for each ITB day) GDSplacebo - GDShome = GDSchangescore2, expressed in % (calculated for the mean of the two placebo days) Criteria for being a candidate for pump implantation: a greater than or equal to 25% difference between the GDSchangescore1 and GDSchangescore2 present on two subsequent days (responder). Implantation phase: After the screening phase a programmable pump (SynchroMed Infusion system, Medtronic INC, Minneapolis MN) for continuous ITB administration will be implanted in patients who fulfill the criteria stated above. During this phase ITB therapy will be started at a dose double the effective screening dose and will be titrated for a maximum effect over a three-month period. All implanted patients will be co-managed by the department of rehabilitation. Following implantation, severely affected patient will be referred to an in-patient rehabilitation unit. Mild to moderately affected patients will be seen in the out-patient rehabilitation unit. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Intrathecal baclofen (ITB) |
| Primary outcome measure | 1. GDS will be calculated separately at baseline (GDShome) and during a week at one year of follow-up. The difference between these two measurements is identified as the change from baseline in GDS at one-year follow-up (GDS1year) 2. Dystonia related Functional Limitations (DFL) are self-assessed at hourly intervals across the day using four items, addressing upper extremity function, capability of making transfers and mobility. Each item is assessed on a zero to three scale. DFL will be calculated at baseline (DFLhome) and during a week at one year of follow-up. The difference between these two measurements is identified as the change from baseline in DFL at one year follow-up (DFL1year). |
| Secondary outcome measures | 1. RSD related impairments 2. Activities of Daily Living (ADL) and quality of life will be assessed separately before implantation and at one-year follow-up The difference between these two measurements is identified as the change from baseline for each score. |
| Overall study start date | 01/01/2002 |
| Completion date | 01/01/2006 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Not Specified |
| Sex | Not Specified |
| Target number of participants | 45 |
| Key inclusion criteria | 1. All patients should fulfill the diagnostic criteria of the complex regional pain syndrome consensus report of the International Association for the Study of Pain (IASP): 1.1. Continuing pain, allodynia or hyperalgesia, in which the pain is disproportionate to any inciting event 1.2. Evidence at some time of oedema, changes in skin blood flow, or abnormal sudomotor activity in the region of the pain 1.3. No condition that would otherwise account for the degree of pain and dysfunction 2. All patients must suffer from tonic dystonia in one or more extremities, that may cause fixed postures at rest of variable severity 3. Before starting the study all patients will have received a trial with oral baclofen. Only patients with an insufficient response or dose-limiting sedative effects to oral baclofen are eligible for this study |
| Key exclusion criteria | Does not comply with the above inclusion criteria |
| Date of first enrolment | 01/01/2002 |
| Date of final enrolment | 01/01/2006 |
Locations
Countries of recruitment
- Netherlands
Study participating centre
Leiden University Medical Centre (LUMC)
Leiden
2300 RC
Netherlands
2300 RC
Netherlands
Sponsor information
Leiden University Medical Centre (LUMC) (The Netherlands)
Hospital/treatment centre
Hospital/treatment centre
Department of Neurology
P.O. Box 9600
Leiden
2300 RC
Netherlands
| Website | http://www.lumc.nl/english/start_english.html |
|---|---|
"ROR" |
https://ror.org/027bh9e22 |
Funders
Funder type
Industry
Leiden University Medical Centre (LUMC) (The Netherlands)
No information available
Medtronic Europe S.A. (Switzerland)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
